Study: Denosumab Effective in Treating Osteoporosis in Transfusion Dependent Thalassemia
October 31, 2018) — For patients with osteoporosis caused by
transfusion-dependent thalassemia (TDT), a twice-yearly injection appears to
improve spinal bone mineral density, according to a new study.
study, published online today in the journal Blood
Advances, also suggests that this treatment, denosumab, could
reduce pain and improve quality of life for people with TDT and osteoporosis.
Background: Osteoporosis is a bone disease that
affects 40 percent of people with TDT — an inherited congenital blood disorder
that results in the decreased production of hemoglobin and red blood cells — and
is one of the most prevalent comorbidities associated with the disorder. People
with osteoporosis have porous, weak bones that are susceptible to fractures and
often cause pain. Current standard therapy for people with TDT and osteoporosis
is to administer intravenous bisphosphonate agents such as pamidronate or
It is believed that
people with thalassemia and osteoporosis exhibit elevated levels of the
osteoporosis regulator gene known as RANKL. Denosumab, delivered under the skin
instead of intravenously, is an FDA-approved anti-RANKL therapy that is not yet
approved for use in people with TDT-induced osteoporosis.
Study methods: Researchers
established a single-site, randomized, placebo-controlled, double blind phase IIb
trial to evaluate the efficacy and safety of denosumab in patients with TDT and
osteoporosis. 63 patients were randomly assigned to receive either 60mg of
denosumab (n=32) or placebo (n=31) on days 0 and 180 over a period of 12
months. Each patient was also provided with daily supplements of calcium and
vitamin D throughout the study.
Results: Each patient’s
bone mineral density was measured in the L1–L4 lumbar spine, the wrist, and the
femoral neck. Patients receiving denosumab had a 5.92 percent increase in lumbar
bone density compared to a 2.92 percent increase in the placebo arm. Similarly,
patients receiving denosumab lost less bone mineral density in their wrist
compared to placebo (-0.26% vs -3.92%, respectively), while increasing density
in the femoral neck (4.008% and 1.96%, respectively).
patients receiving denosumab reported a significant reduction in pain compared
to those in the placebo arm.
only is denosumab associated with improved bone health and reduced pain, but
its ease of administration may very well make this drug superior to
bisphosphonates for the treatment of osteoporosis in patients with TDT and
osteoporosis,” said senior study author Evangelos Terpos, MD, of the National
and Kapodistrian University of Athens, Greece.
serious adverse events were reported in the denosumab arm, including pleural
effusion, supraventricular tachycardia, and atrial fibrillation, though
investigators found no link between the treatment and these adverse events.
Conclusion: This study suggests
that future studies should directly compare denosumab to bisphosphonates for
the treatment of bone mineral density loss and pain management for people with
TDT and osteoporosis.
Additional Notes: This study was
funded by Amgen.
Blood Advances (www.bloodadvances.org), is a peer-reviewed, online only, open access
journal of the American Society of Hematology (ASH) (www.hematology.org), the world’s largest professional society concerned
with the causes and treatment of blood disorders.
ASH’s mission is
to further the understanding, diagnosis, treatment, and prevention of disorders
affecting blood, bone marrow, and the immunologic, hemostatic, and vascular
systems by promoting research, clinical care, education, training, and advocacy
Blood Advances® is
a registered trademark of the American Society of Hematology.
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Szabo, American Society of Hematology